UK approves drug for the treatment of 2 different blood diseases
In the UK, a drug utilizing a gene therapy method that aims to treat 2 different hereditary blood diseases by editing the causative gene has been approved.
AA
In a statement issued by the UK's Medicines and Healthcare products Regulatory Agency (MHRA), it was stated that the drug named "Casgevy," which utilizes the gene-editing technology CRISPR, has been approved for use in the treatment of sickle cell disease and beta-thalassemia.
FIRST LICENSED DRUG USING CRISPR
The statement noted that Casgevy is the first licensed drug to use CRISPR, also known as "genetic scissors," which was developed by 2 scientists who won the 2020 Nobel Prize in Chemistry and allows for the editing of genes by cutting them on DNA.
The statement, which conveyed that Casgevy, which utilizes a gene therapy method, could eliminate the pain, infection, and anemia caused by these 2 diseases, included the following:
"Casgevy is designed to edit the faulty gene in the patient's bone marrow stem cells to enable the body to produce functional hemoglobin. To do this, stem cells are taken from the bone marrow, edited in a laboratory environment, and then re-infused into the patient's body."